"The strategic restructuring brings enhanced focus to our pipeline, streamlines operations and improves our financial strength," stated Matthew Kapusta, interim chief executive officer of uniQure. "We are committed to the timely development of our programs in hemophilia B and Huntington's disease, as well as the support of our collaboration with BMS. Along with our investigators and collaboration partner, we are enthusiastic about the interim data from our ongoing Phase I/II study of AMT-060 in hemophilia B and will allocate necessary resources to expedite bringing AMT-060 to market, with commercial manufacturing capabilities in our state-of-the-art U.S. facility which is already in place.""We are confident that these efforts will significantly streamline operations and position us well for the future," Mr. Kapusta added. "We are grateful for the contributions that the employees affected by this outcome have made to uniQure, and we will be supporting them throughout this transition." Key Outcomes of the Strategic Review Pipeline Prioritization uniQure has decided to prioritize the following programs within its gene therapy pipeline:
- Pursue as top priorities the clinical development of its product candidates in hemophilia B and Huntington's disease. The Company intends to initiate a pivotal trial in hemophilia B pending discussions with regulatory authorities and to file an investigational new drug (IND) application for Huntington's disease following the completion of ongoing IND-enabling studies.
- Provide greater focus on new product generation, with an emphasis on liver-directed diseases. This includes the internal development or in-license of new product candidates in rare and orphan diseases that can leverage the Company's next-generation vector and promoter platform and manufacturing capabilities.
- Advance uniQure's collaboration with BMS in cardiovascular diseases as a top research priority, specifically focused on the advancement of S100A1 in congestive heart failure.
- Evaluate options for its gene therapy programs targeting Sanfilippo B and Parkinson's disease, including partnering opportunities. Two of the four patients in the ongoing Phase I/II clinical trial in Sanfilippo B have completed their 30-month evaluations, and the 30-month data on all four patients in the study are expected to be available for presentation in the first quarter of 2017. Regarding the academic-sponsored trial in Parkinson's disease, uniQure will evaluate partnership opportunities to accelerate completion of the ongoing study and further develop the program.
- uniQure will consolidate all GMP manufacturing at its Lexington, MA facility. The Lexington facility is fully operational and has the capability of scaling up to 2,000L capacity, which uniquely positions the Company for late-stage development and commercialization of its gene therapy products.
- The Company will maintain a smaller, but fully integrated research and development organization in Amsterdam, the Netherlands. The team is expected to move into a new facility in the first half of 2017.
- The previous organizational structure based on therapeutic areas of focus is being eliminated. As a result of this change, Deya Corzo, M.D., senior vice president of the liver and metabolic therapeutic area, and Charles Richard, M.D., Ph.D., senior vice president of the central nervous system therapeutic area are transitioning their responsibilities and will be leaving the Company by the end of the year.
- Christian Meyer, M.D., Ph.D., chief medical officer, will assume full responsibility for advancing the company's hemophilia B gene therapy program.
- Harald Petry, Ph.D., chief scientific officer, will continue to lead uniQure's preclinical research, including IND-enabling studies in Huntington's disease.
Conference Call InformationuniQure will host a conference call today, November 15, 2016 at 8:30 a.m. ET to discuss this announcement. To access the live call by phone, dial (877) 280-2296 (United States) or +44 (0)20 3427 1917 (international); the conference ID is 4243017. The call may also be accessed through the Investors section of the Company's website at www.uniQure.com. Following the live webcast, a replay of the call will be available at the same location through November 30, 2016. About uniQure uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington's disease and cardiovascular diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to," "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding the implementation and effects of the Company's new strategic and organizational changes, the development of our gene therapy product candidates, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with corporate reorganizations and strategic shifts, collaboration arrangements, our and our collaborators' clinical development activities, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's 2015 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2016. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: Maria E. CantorDirect: 339-970-7536Mobile: 617-680-9452 m.cantor@uniQure.com Tom MaloneDirect: 339-970-7558Mobile: 339-223-8541 t.malone@uniQure.com Eva MulderDirect: +31 20 240 6103Mobile: +31 6 52 33 15 79 e.mulder@uniQure.com PDF Version: http://hugin.info/157414/R/2056691/770399.pdf